GB201522416D0 - Wilson's disease gene therapy - Google Patents

Wilson's disease gene therapy

Info

Publication number
GB201522416D0
GB201522416D0 GBGB1522416.5A GB201522416A GB201522416D0 GB 201522416 D0 GB201522416 D0 GB 201522416D0 GB 201522416 A GB201522416 A GB 201522416A GB 201522416 D0 GB201522416 D0 GB 201522416D0
Authority
GB
United Kingdom
Prior art keywords
wilson
gene therapy
disease gene
disease
therapy
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
GBGB1522416.5A
Other languages
English (en)
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
Original Assignee
UCL Business Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by UCL Business Ltd filed Critical UCL Business Ltd
Priority to GBGB1522416.5A priority Critical patent/GB201522416D0/en
Publication of GB201522416D0 publication Critical patent/GB201522416D0/en
Priority to PCT/GB2016/053989 priority patent/WO2017103624A1/fr
Priority to ES16816375T priority patent/ES2862164T3/es
Priority to EP16816375.6A priority patent/EP3390623B1/fr
Priority to US16/063,451 priority patent/US11077208B2/en
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Environmental Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
GBGB1522416.5A 2015-12-18 2015-12-18 Wilson's disease gene therapy Ceased GB201522416D0 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
GBGB1522416.5A GB201522416D0 (en) 2015-12-18 2015-12-18 Wilson's disease gene therapy
PCT/GB2016/053989 WO2017103624A1 (fr) 2015-12-18 2016-12-19 Thérapie génique de la maladie de wilson
ES16816375T ES2862164T3 (es) 2015-12-18 2016-12-19 Genoterapia de la enfermedad de Wilson
EP16816375.6A EP3390623B1 (fr) 2015-12-18 2016-12-19 Thérapie génique pour la maladie de wilson
US16/063,451 US11077208B2 (en) 2015-12-18 2016-12-19 Wilson's disease gene therapy

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1522416.5A GB201522416D0 (en) 2015-12-18 2015-12-18 Wilson's disease gene therapy

Publications (1)

Publication Number Publication Date
GB201522416D0 true GB201522416D0 (en) 2016-02-03

Family

ID=55311248

Family Applications (1)

Application Number Title Priority Date Filing Date
GBGB1522416.5A Ceased GB201522416D0 (en) 2015-12-18 2015-12-18 Wilson's disease gene therapy

Country Status (5)

Country Link
US (1) US11077208B2 (fr)
EP (1) EP3390623B1 (fr)
ES (1) ES2862164T3 (fr)
GB (1) GB201522416D0 (fr)
WO (1) WO2017103624A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210369870A1 (en) 2018-11-16 2021-12-02 Encoded Therapeutics, Inc. Compositions and methods for treating wilson's disease
CN111088285B (zh) * 2019-08-15 2021-12-28 北京锦篮基因科技有限公司 携带atp7b基因表达框及变异体的aav载体及应用
WO2022187571A1 (fr) 2021-03-04 2022-09-09 Research Institute At Nationwide Children's Hospital Produits et procédés de traitement de myopathies à base de dystrophine utilisant crispr-cas9 pour corriger les duplications d'exon dmd
CN117947035A (zh) * 2024-03-25 2024-04-30 上海凌医生物科技有限公司 一种增强基因表达的启动子

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2108927C (fr) 1993-09-21 2008-09-02 Peter Bull Gene de la maladie de wilson
DE10156121A1 (de) * 2001-11-16 2003-05-28 Wolfgang Stremmel Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
WO2012121324A1 (fr) 2011-03-08 2012-09-13 国立大学法人徳島大学 Méthode de sélection d'un groupe efficace de traitements anticancéreux combinant l'utilisation de trois agents, à savoir un agent anticancéreux à base de taxane, un agent anticancéreux à base d'un complexe de platine et un agent anticancéreux à base de fluorure de pyrimidine
KR20130098749A (ko) 2012-02-28 2013-09-05 사회복지법인 삼성생명공익재단 상염색체 열성질환 진단용 키트
CN103224556B (zh) 2013-01-31 2015-04-15 清华大学 亨廷顿舞蹈症的靶位蛋白质及其编码基因和应用
MX2017007965A (es) 2014-12-17 2018-01-18 Fundacion Para La Investig Medica Aplicada Construcciones de acido nucleico y vectores de terapia de genes para uso en el tratamiento de enfermedad de wilson.
DK3233129T3 (da) 2014-12-17 2020-04-06 Fundacion Para La Investig Medica Aplicada Nukleinsyrekonstrukter og genterapivektorer til anvendelse i behandlingen af wilsons sygdom og andre lidelser

Also Published As

Publication number Publication date
EP3390623B1 (fr) 2021-02-17
ES2862164T3 (es) 2021-10-07
US20200268904A1 (en) 2020-08-27
US11077208B2 (en) 2021-08-03
EP3390623A1 (fr) 2018-10-24
WO2017103624A1 (fr) 2017-06-22

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Legal Events

Date Code Title Description
AT Applications terminated before publication under section 16(1)